Agios Pharmaceuticals (NASDAQ:AGIO) announced today that the U.S. Food and Drug Administration (FDA) has approved AQVESME™ (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia.
The decision makes AQVESME the first oral, disease-modifying medicine authorized for the full spectrum of the disease, covering both non-transfusion-dependent (NTD) and transfusion-dependent (TD) patients.
The approval fills a critical gap for approximately 6,000 adult patients in the U.S. living with thalassemia, a rare blood disorder that causes the body to produce an abnormal form of hemoglobin.
While the drug is already marketed as PYRUKYND® for pyruvate kinase (PK) deficiency, Agios will launch the thalassemia indication under the brand name AQVESME in late January 2026.
This rebranding is tied to a specific Risk Evaluation and Mitigation Strategy (REMS) mandated by the FDA to manage potential liver safety risks.
The FDA's authorization was based on the Phase 3 ENERGIZE and ENERGIZE-T trials, which collectively enrolled 452 patients.
The studies demonstrated that by activating the pyruvate kinase enzyme, the drug increases ATP production, which in turn improves red blood cell health and survival.